Presentation (CTI)

OBJECTIVES: HTA agencies typically prefer utility values from generic measures, but this can be challenging for rare diseases due to small, heterogenous populations, and measures which may not sufficiently capture impact on HRQoL. Where utility values from clinical trials are unavailable or generic measures e.g. EQ-5D are insufficiently sensitive for HRQoL assessment, vignette studies can be employed. We therefore sought to investigate the acceptability of vignette studies to elicit utility values in rare diseases to inform HTA submissions and any methodological considerations given the criticality of utility data in HTA submissions.
METHODS: HTA guidance documents were reviewed for eight European countries, for guidance on use of vignette-based methodology. Countries were ranked by level of acceptability.
RESULTS: NICE in the UK, and HTA agencies which reference NICE (Denmark and Norway), acknowledge that direct patient elicitation is preferred (e.g. through generic utility measures); however, where data are unavailable or infeasible, vignette-based approaches are acceptable if rigorously conducted. HTA agencies in France, Italy, Sweden, and Spain are pragmatic, but require strong justification and transparency. There was a lack of specific guidance for use of vignette-based methods in Italy and Spain. German HTA had relatively low levels of acceptability and strong preference for the EQ-5D or empirical patient data.
CONCLUSIONS: European HTA agencies express a preference for direct patient-reported utility data but may accept vignette studies for rare diseases if rigorous methods are used. This is critical given the challenges of demonstrating value in rare diseases and that generic measures can have limited applicability and sensitivity in these populations. Given the impact that the utility data can have on the resulting ICER and assessment of cost-effectiveness, consideration of how to generate the appropriate utility data whilst adhering to rigorous data collection methods is vital in optimising patient access to new therapies.